First positive regulatory agency determination for a PCSK9 InhibitorNews - May 25, 2015
The CHMP recommended granting evolocumab marketing authorization for:
- The treatment of adults with primary hypercholesterolemia (heterozygous familial and non-familial [HeFH]) or mixed dyslipidemia, as an adjunct to diet:
- in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin, or
- alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.
- The treatment of adults and adolescents aged 12 years and over with homozygous familial hypercholesterolemia (HoFH) in combination with other lipid-lowering therapies.
The effect of evolocumab on cardiovascular morbidity and mortality has not yet been determined.
"We are pleased to receive a positive opinion from the CHMP for evolocumab as it is an important step in providing a new treatment option for patients with high cholesterol, who are unable to reach their LDL cholesterol goals with current therapies in the European Union," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Uncontrolled high cholesterol is a burden on the health system and we look forward to continuing to work with regulatory authorities to bring evolocumab to patients across Europe." The Icelandic and Norwegian CHMP members have also agreed with the above-mentioned recommendation on granting of the marketing authorization.
The CHMP positive opinion will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, a centralized marketing authorization with unified labeling will be granted in the 28 countries that are members of the EU. Norway, Iceland and Liechtenstein, as members of the European Economic Area (EEA), will take corresponding decisions on the basis of the decision of the EC.
The CHMP opinion is based on data from approximately 6,800 patients, including more than 4,500 patients with high cholesterol in 10 Phase 3 trials. The Phase 3 studies evaluated the safety and efficacy of evolocumab in patients with elevated cholesterol, including patients on statins with or without other lipid-lowering therapies; patients who cannot tolerate statins; patients with HeFH; and patients with HoFH.
Press release Amgen May 22 2015